Katagiri, Yasuhiro (NHLBI)
- In vitro data available
- In vivo data available (animal)
The invention is directed to modification of a particular sugar by the enzyme arylsulfatase B (ARSB), which results in axon regeneration.
Following traumatic brain or spinal cord injury, glial scars prevent regeneration of axons. Chondroitin sulfate proteoglycans (CSPGs) are major components of glial scars. CSPGs are made of a protein core containing glycosaminoglycan (GAG) sugar side chains, which, when sulfated, are responsible for the inhibitory activity of glial scars. Specifically, NIH researchers have shown that the 4-sulfate unit on a certain sugar on GAG is responsible for inhibiting axon regrowth and, when the 4-sulfate unit is reduced, axon regrowth is observed. Moreover, removal of this 4-sulfate unit by the ARSB enzyme promotes axon regrowth.
As a potential therapy for spinal cord injuries, researchers developed a vector expressing ARSB and demonstrated that this vector promotes axon regeneration when injected into the spinal cord of a mouse.
- Treatment of brain and spinal cord injury
- Treatment of other CNS injuries, including stroke
- Treatment of heart attack
- There are no existing products for treatment of traumatic spinal cord injury
- ARSB is already approved for treatment of Mucopolysaccharoidosis VI, a lysosomal storage disease